Advanced Biotherapy Regulatory Considerations

Patrick Bedford, VP, regulatory affairs & development at Artisan Biotechnologies, discusses the challenges of meeting regulatory requirements in advanced therapies.

GEN: What are the key trends in regulation for advanced therapies?

Patrick Bedford: Cell and gene therapies face regulatory uncertainty and investors are concerned, as they’re unsure if companies can meet the regulator’s expectations. That’s not abnormal with emerging technologies and uncertainty isn’t always a bad thing. Companies should now be front-loading risk assessments and not rushing into the clinic.

GEN: What are the challenges and opportunities of published regulatory guidance?

Bedford: People always complain guidance documents don’t specifically address their needs, but they’re written to a common denominator. Because they’re not super-specific, you have flexibility to interpret guidance to competitive advantage.

GEN: Where are the gaps in applying generalized regulations to process development?

Bedford: There’s FDA draft guidance for gene editing but it is missing important points of reference. If you’re using in silico prediction algorithms, you can put different parameters for how many mismatches are allowed. It’s unclear how to justify how many mismatches you allow, and there’s a disincentive to identify too many hazards as you need to show you’ve addressed these.

GEN: What are the main differences between regulatory requirements in different countries?

Bedford: I’m familiar with the United States and Canada. There’s been a concentrated effort to harmonize policies and regulatory requirements between jurisdictions. The EMEA, FDA, Health Canada, and PMDA tend to meet every three months to discuss harmonization from a policy perspective, for example. As such, differences tend to come down to individual reviewers more than the jurisdiction.

GEN: How should advanced therapy companies manage the evolving regulatory environment as they move through drug development?

Bedford: I’ve broken this down into four steps:

• I’d start by reviewing guidance that’s out there that may be relevant, starting with ICH guidelines then moving on to your jurisdiction of interest. This includes guidance for biologics and will increasingly include guidance documents for cell and gene therapy in the future.
• Understand the guidance documents and how they’re relevant and develop your own risk-based regulatory strategy.
• Participate in the development of regulatory policy to ensure you’re at the cutting edge of their development.
• Finally, approach the regulator before making significant investments in your technology so that you can move forward with your development program with confidence.

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